Resumen de: US20260027178A1
The present disclosure generally relates to the treatment of subjects having Parkinson's disease or at risk of developing Parkinson's disease by administering a SIM BHLH Transcription Factor 2 (SIM2) agonist to the subject.
Resumen de: US20260027234A1
The present disclosure provides recombinant vectors encoding a choline acetyltransferase (ChAT) polypeptide, compositions thereof, and methods of use thereof.
Resumen de: US20260028623A1
Provided are RNAi agents, pharmaceutical compositions, and methods for reducing the amount or activity of PRNP RNA in a cell or a subject, and in certain instances reducing the amount of prion protein in a cell or a subject. Such RNAi agents, pharmaceutical compositions, and methods are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such neurodegenerative diseases include prion diseases, such as Creutzfeldt-Jakob disease (CJD) (e.g., variant Creutzfeldt-Jakob Disease (vCJD), classic Creutzfeldt-Jakob Disease (cCJD), familial Creutzfeldt-Jakob Disease (fCJD), or sporadic Creutzfeldt-Jakob Disease (sCJD)), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, or kuru; synucleinopathies such as Alzheimer's disease, Parkinson's disease, or dementia with Lewy bodies; or tauopathies such as frontal temporal dementia associated with a Tau mutation, Pick's disease, progressive supranuclear palsy, corticobasal neurodegeneration, or chronic traumatic encephalopathy (CTE).
Resumen de: US20260028625A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Resumen de: US20260028347A1
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
Resumen de: WO2026024901A1
The present disclosure provides methods of using a small molecule TREM2 agonist or a pharmaceutically acceptable salt thereof to treat Alzheimer's Disease in a human subject.
Resumen de: WO2026024717A1
Provided herein are methods and compositions for treating neurological diseases (e.g., neurodegenerative disorders (e.g., Alzheimer's disease, Parkinson's disease, dementia, a tauopathy, chronic traumatic encephalopathy (CTE), traumatic brain injury (TBI), mild cognitive impairment); psychiatric disorders (e.g., bipolar disorder, schizophrenia, depression, anxiety, post-traumatic stress disorder, obsessive compulsive disorder); inflammation in the central nervous system) using lithium salts, activators that increase the expression or activity of the RE1 silencing transcription factor (REST), or a combination thereof, or in combination with an additional agent.
Resumen de: WO2026024812A1
The present disclosure provides substituted 2,3,4,5-tetrahydro-1H-benzo e 1,4 diazepin-l-yl compounds, pharmaceutically acceptable salts thereof, or a pharmaceutical composition thereof, utilized in the manufacture of a medicament for the inhibition of F-ATP hydrolase and for treating diseases, disorders or conditions associated with F-ATP hydrolase, including Alzheimer's disease, Parkinson's Disease, amyotrophic lateral sclerosis, Friedreich's ataxia and cancer.
Resumen de: EP4685158A1
The present invention relates to the field of neurodegenerative diseases, in particular Alzheimer's disease. The present invention further relates to fibrillary Apolipoprotein E (ApoE) for use in a method of treatment and/or prevention of a neurodegenerative disease and methods of producing said fibrillary ApoE. Moreover, the present invention relates to an antigen-binding peptide specifically binding to fibrillary ApoE, preferably human ApoE, a method of generating said antigen-binding peptide, and its use in a method of treatment and/or prevention and/or diagnosis of a neurodegenerative disease in a patient in need thereof.
Resumen de: AU2024238511A1
The present disclosure relates to biomarkers and uses thereof in methods for selecting a patient diagnosed with amyotrophic lateral sclerosis (ALS) for an ALS therapy. The present disclosure further relates to methods for identifying the severity of ALS in a patient, treating an ALS patient, and monitoring efficacy of an ALS treatment.
Resumen de: WO2026017171A1
The present invention relates to a heterocyclic carbonyl derivative modulator, a preparation method therefor and a use thereof. In particular, the present invention relates to a compound represented by general formula (VIII-1A), a preparation method therefor, a pharmaceutical composition containing the compound, and a use of the compound as a modulator in the treatment of Alzheimer's disease, schizophrenia, pain, addiction, and sleep disorders. Each substituent in general formula (VIII-1A) is the same as defined in the description.
Resumen de: WO2026017114A1
Use of a phenylpyrimidinone compound. The present invention specifically relates to use of a phenylpyrimidinone compound represented by formula (I) or a tautomer thereof, a pharmaceutically acceptable salt thereof, a solvate thereof, or an isotopically labelled compound thereof in the preparation of a drug for ameliorating or treating cognitive impairment, and/or ameliorating or treating cerebrovascular diseases. Studies using cell and animal models have shown that compared to the commercially available clinical drug donepezil and the investigational clinical drug mirodenafil, the compound has demonstrated significant therapeutic characteristics and substantial technical advancements in terms of protecting nerve cells, ameliorating cognitive impairment, ameliorating cerebrovascular diseases, and ameliorating degenerative diseases such as Parkinson's disease.
Resumen de: WO2026018132A1
Provided is a method for treating a condition selected from the group consisting of pain, sleep disorder, cancer, Alzheimer's disease, neurodegenerative diseases, ulcer, hypertension, opioid- related adverse effects, inflammation, asthma, glaucoma, neurodevelopment diseases and combinations thereof in a subject in need thereof, the method comprising administering to the subject: a) a first active pharmaceutical ingredient (API) comprising at least one cannabinoid other than CBD and THC; and b) a second API, which is different from the first API.
Resumen de: WO2026016523A1
The use of the traditional Chinese medicine (TCM) components to manufacture the medicament for diminishing effects of Amyotrophic Lateral Sclerosis (ALS) or neurodegeneration in a predisposed subject, wherein the TCM comprising: (1) Ge Gen, (2) Dang Gui, (3) Dan Shen, (4) Dang Shen, (5) Huang Qi, (6) Zi Su Zi, (7) Da Zao, (8) Chai Hu, (9) Huang Qin, (10) Hong Hua, (l l) Yu Jin, (12) Da Huang, (13) Hua Jiao, (l4) Gan Cao, (15) Mai Dong, (l6) Wu Wei Zi, (17) Fu Zi, (18) Ren Shen, (l9) Fu Ling, (20) Shi Gao, (21) Mu Li, (22) Gui Zhi, and (23) Che Qian Zi; or, extracts thereof in amounts equivalent to the amounts of the raw materials of the group of ingredients. The patent is targeted on the clinical efficacy of the different pathological forms of ALS disease manifestation. The medicament could be used in a combination with a Western medicine.
Resumen de: WO2026018015A2
The present invention relates inter alia to methods for identifying a substance useful for the prevention or treatment of a disease, disorder, or condition associated with altered NLRX1 activity, and uses of said substances in the prevention or treatment of a disease, disorder, or condition associated with altered NLRX1 activity, for example in the prevention or treatment of Parkinson's disease or amyotrophic lateral sclerosis.
Resumen de: WO2026018824A1
The purpose of the present invention is to provide a drug for treating or preventing neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). The present inventors identified oligonucleotides that inhibit MP-13 mRNA, which is a splicing variant (TDPsv) of TDP-43. Use of these oligonucleotides can reduce the amount of MP-13 mRNA and suppress protein expression. These oligonucleotides can be useful for the treatment or prevention of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).
Resumen de: WO2026019699A1
The present disclosure is directed to engineered cells designed to sense tau and to express one or more proteins in response to this binding event. In addition, the cells and associated methods of use can detect, treat, and recapitulate the symptoms of Alzheimer's disease. The engineered cells can regulate expression of neuronal growth factors and anti-inflammatory proteins to address neurodegeneration and neuroinflammation, respectively.
Resumen de: WO2026020097A1
Provided herein are postbiotic compositions prepared using successive fermentation methods of specific bacteria and plant fiber material. Also provided herein are methods, including for the treatment or prevention of the disruption of gut microbiota, or dysbiosis, associated with an antibiotic treatment, chemotherapy treatment, or administration of a dysbiosis-causing medications or medical treatments, using said postbiotic compositions, or for improving responses and/or reducing complications to treatments such as antibiotics or chemotherapy. Such postbiotic compositions can also be used for the treatment or prevention of the disruption of the gut-brain axis, including to treat or prevent neurological diseases or disorders, such as synucleinopathies, including but not limited to Parkinson's disease.
Resumen de: WO2026020153A1
Composition and methods for treating neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Angelman's Syndrome (AS), the compositions specifically including antisense oligonucleotides (ASOs) containing thiomorpholino nucleotides configured to inhibit ribosomal frameshifting of paternally expressed gene 10 (PEG 10) mRNA during translation, thereby inhibiting the formation of the long form gag-pol protein.
Resumen de: WO2026019879A1
The general field of the present disclosure are novel approaches to the treatment of Alzheimer's and other neurodegenerative disorders using novel therapeutics comprising Lyn kinase inhibitors. Specifically, the disclosure provides compounds, pharmaceutical compositions, and methods of treating and preventing such disorders.
Resumen de: US20260023070A1
Provided herein are methods and compositions for identifying α-synuclein aggregation inhibitors. Also provided are methods of use of the α-synuclein aggregation inhibitors; the methods include methods of inhibition the formation of Lewi bodies and methods of treating synucleinopathies in subjects. Methods are compositions provided herein include optogenetic α-synuclein fusion proteins and an optogenetic alpha-synuclein (α-syn) aggregation system. Further, provided herein are compositions comprising α-synuclein aggregation inhibitor drug candidates identified using an optical alpha-synuclein aggregation screening system. The α-synuclein aggregation inhibitor drug candidates have neuroprotective effects in vitro and in vivo and provide proof-of principle that the optical alpha-synuclein aggregation screening system can be used to identify drug candidate for synucleopathies and tauopathies, including for example Parkinson's disease.
Resumen de: US20260021100A1
The present invention provides methods for reducing amyloid beta formation and for treating diseases associated with the accumulation of amyloid beta. The present invention provides (1) A β aggregation inhibition by A β Oligomer/Fibril formation inhibition, (2) BACE-1 reduction through β-Amyloidogenic Processing inhibition, (3) increased cerebral blood flow, (4) activation of Neuronal cell Death inhibition and Neurogenesis, Synaptogenesis, Angiogenesis promotion, (5) DKK-1 inhibition by Wnt Signaling and Aβ production Positive Feedback Loop for inhibition of APP to suppress Aβ accumulation, (6) Autophagy activation by cells, by providing Mirodenafil, Sildenafil, Vardenafil, Tadalafil, Udenafil, Dasantafil, and Avanafil and a Pharmaceutically Acceptable Salt, Solvate, and Hydrate in selected compounds key of ingredient containing drug compound composition, and this with the treatment method provided.
Resumen de: US20260021150A1
Disclosed is a method for diminishing effects of Amyotrophic Lateral Sclerosis (ALS) in administering to the predisposed subject effective amounts of Traditional Chinese Medicine (TCM) or in a combination of a Western medicine, wherein the TCM including: (1) Ge Gen, (2) Dang Gui, (3) Dan Shen, (4) Dang Shen, (5) Huang Qi, (6) Zi Su Zi, (7) Da Zao, (8) Chai Hu, (9) Huang Qin, (10) Hong Hua, (I I) Yu Jin, (12) Da Huang, (13) Hua Jiao, (I4) Gan Cao, (15) Mai Dong, (I6) Wu Wei Zi, (17) Fu Zi, (18) Ren Shen, (I9) Fu Ling, (20) Shi Gao, (21) Mu Li, (22) Gui Zhi, and (23) Che Qian Zi; or, extracts thereof in amounts equivalent to the amounts of the raw materials of the group of ingredients. The patent is targeted on the clinical efficacy of the different pathological forms of ALS disease manifestation.
Resumen de: US20260021089A1
An application method of brucine for treating amyotrophic lateral sclerosis (ALS) is provided, relating to the field of biomedicines. It can delay a time of onset of ALS, prolong survival time of ALS patients, delay a weakening of limb extension ability, and improve limb grip strength.
Nº publicación: US20260022376A1 22/01/2026
Solicitante:
THE TRUSTEES OF INDIANA UNIV [US]
The Trustees of Indiana University
Resumen de: US20260022376A1
The present disclosure provides novel approaches to the treatment of Alzheimer's disease, and other neurodegenerative disorders such as chronic traumatic encephalopathy (CTE) using novel therapeutics comprising agents that reduce the interaction of a tau seed interactor with intracellular tau proteins and thus reduce or inhibit the production of tau-associated neurofibrillary tangles.